E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cerebral Cavernous Malformation (CCM) |
Malformazione Cavernosa Cerebrale (CCM) |
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E.1.1.1 | Medical condition in easily understood language |
The term refers to a familial decease characterized by mulberry-shaped abnormal blood vessels in the brain or spine. |
Una patologia familiare, caratterizzata da malformazioni vascolari (angiomi) nel cervello o nel midollo spinale. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10071747 |
E.1.2 | Term | Cerebral cavernous malformation |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Test whether a chronic treatment with propranolol will reduce the burden of cerebrovascular lesions, of clinical events symptoms in patients with familial CCM |
Valutare l'efficacia del propranololo nella riduzione delle lesioni cerebrovascolari e degli eventi clinici nei pazienti CCM familiare. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Patients with Familial cerebral cavernous malformations (FCCM); 2.history of clinical symptoms or events: intracerebral hemorrhage, stroke, permanent or transient focal deficits, seizures, disability or any other neurological symptom supposedly related to CCM; 3.age of at least 18 years. 4.Written informed consent to participate in the study prior to any study procedures.
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1.Pazienti CCM familiare sintomatica; 2.storia clinica con sintomatologia CCM o eventi clinici: emorragia intracerebrale, ictus, deficit focale permanente o transitorio, epilessia, disabilità o qualsiasi altro sintomi neurologici associati a CCM; 3.individui adulti (> 18 anni); 4.consenso informato firmato per la partecipazione allo studio, prima del suo inizio.
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E.4 | Principal exclusion criteria |
1.Implanted pacemaker or any other condition preventing the MRI exam; 2.bradycardia, 2nd or 3rd degree AV block or symptomatic hypotension; 3.unstable diabetes; 4.severe asthma; 5.liver and/or renal failure; 6.current use of verapamil or diltiazem for risk of excessive bradycardia; 7.previous brain surgery (within 6 months); 8.known hypersensitivity to study drug (propranolol or any of the ingredients) 9.pregnant or lactating women or women of childbearing potential who are not protected from pregnancy by an accepted method of contraception 10.participation to another clinical trial; 11.inability to cooperate with the trial procedures.
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1.Pazienti portatori di pacemaker o qualsiasi altra condizione incompatibile con l’esecuzione di una tecnica di immagine basata sulla risonanza magnetica; 2.bradicardia o blocco arterioventricolare di 2° o 3° grado, ipotensione sintomatica; 3.diabete instabile; 4.asma bronchiale grave; 5.insufficienza epatica o renale; 6.indicazione per verapamil e diltiazem; 7.ipersensibilità nota al farmaco in sperimentazione (propranololo e/o suoi eccipienti); 8.chirurgia cerebrale pregressa (<6 mesi); 9.gravidanza e allattamento; donne in età fertile non protette da misure anticoncezionali adeguate; 10.partecipazione ad un altro studio clinico; 11.incapacità a seguire le procedure dello studio.
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E.5 End points |
E.5.1 | Primary end point(s) |
New occurrence of clinical events CCM-related, that is intracerebral hemorrhage (ICH) and focal neurological deficits (FND) including seizures. |
incidenza di nuovi eventi clinici correlati con la CCM come emorragia intracerebrale e/o deficit neurologici focali, incluse le crisi epilettici. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Microvascular hemorrhages as assessed by brain MRI.; Clinical outcomes such as global cognitive function, global disability and health related quality of life.; Different vascular lesion characteristics such as diameter, length, and location assessed by MRI. |
Emorragie micro-vascolari, identificate nel tessuto cerebrale con MRI. ; Funzione cognitiva globale, disabilit¿ globale e qualit¿ di vita.; Caratteristiche delle diverse malformazioni vascolari (diametro, lunghezza e sede) valutate con MRI. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
24 months; 24 months; 24 months |
24 mesi; 24 mesi; 24 mesi |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
TERAPIA STANDARD |
STANDARD CARE |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |